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Delivery of the 7-dehydrocholesterol reductase gene to the central nervous system using adeno-associated virus vector in a mouse model of Smith-Lemli-Opitz Syndrome

Smith Lemli Opitz syndrome (SLOS) is an inherited malformation and mental retardation metabolic disorder with no cure. Mutations in the last enzyme of the cholesterol biosynthetic pathway, 7-dehydrocholesterol reductase (DHCR7), lead to cholesterol insufficiency and accumulation of its dehyrdocholes...

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Detalles Bibliográficos
Autores principales:	Pasta, Saloni, Akhile, Omoye, Tabron, Dorothy, Ting, Flora, Shackleton, Cedric, Watson, Gordon
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	Elsevier 2015
Materias:	SI:Therapy
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4559272/ https://www.ncbi.nlm.nih.gov/pubmed/26347274 http://dx.doi.org/10.1016/j.ymgmr.2015.07.006

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